1ST Biotherapeutics joins MJFF LRRK2 gene-targeting program
LITE program brings biotechs, academics together to collaborate
1ST Biotherapeutics said it joined an initiative led by the Michael J. Fox Foundation (MJFF) aimed at advancing therapies that target the LRRK2 gene, a key genetic contributor to Parkinson’s disease.
The initiative, MJFF’s LRRK2 Investigative Therapeutics Exchange (LITE) program, brings together biotech companies and academic research institutions with the shared goal of developing LRRK2-targeting treatments for Parkinson’s. Collaborators in the initiative share data, resources, and expertise as they work to develop drugs and testing targeting LRRK2.
“LITE provides a unique opportunity for us to collaborate with world-class researchers and industry leaders in an open science framework,” Jamie Jae Eun Kim, PhD, founder and CEO of 1st Biotherapeutics, said in a company press release. “This partnership strengthens our commitment to rapidly advancing LRRK2-targeted therapies that could significantly impact the lives of Parkinson’s patients.”
Mutations in the LRRK2 gene are among the most common genetic causes of inherited Parkinson’s. LRRK2 produces an enzyme that helps cells communicate and dispose of waste. When the gene is mutated, the enzyme can become overactive, contributing to the cellular dysfunction and neurodegeneration characteristic of Parkinson’s.
Research has suggested that the LRRK2 gene and its enzyme may be promising targets for Parkinson’s therapies. 1st Biotherapeutics, which is based in South Korea, is one of several companies developing LRRK2-targeting Parkinson’s drugs.
Targeting Parkinson’s pathways
The company is advancing multiple investigational therapies for neurodegenerative diseases. Its lead candidate, FB101, is in a Phase 1 clinical trial (NCT05995782) to treat Parkinson’s and amyotrophic lateral sclerosis (ALS). FB101 targets c-Abl, a kinase implicated in the progression of alpha-synuclein accumulation, a key driver of Parkinson’s. A second investigational therapy, 1ST-104, is in preclinical development and is designed to target both LRRK2 and c-Abl, addressing two critical pathways associated with Parkinson’s.
To facilitate LRRK2 drug development and encourage open science collaboration, MJFF launched LITE in 2024. More than 40 pharmaceutical and biotech companies and academic institutions participate in the project.
“MJFF believes in the power of bringing together the expertise of industry and academia to advance LRRK2 drug development efforts,” said Shalini Padmanabhan, MJFF’s senior vice president of discovery and translational research.
Through LITE, researchers plan to establish best practices for LRRK2-targeting therapy development. In addition to exploring ways to target LRRK2, collaborators will investigate LRRK2 biomarkers for use in future clinical trials.
“We look forward to seeing what our partners can achieve with the cooperation of the LRRK2 Investigative Therapeutics Exchange,” Padmanabhan said.
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